Barriers in Gene Therapy

DNA (33391)

Gene therapy is an advanced science and requires an incredible amount of expertise and knowledge and science before it. The following are a few problems that are currently being encountered and battled with.

Lifespan of treatment

There is a great deal of trouble with keeping the introduced genes stable and long-lasting. It is very difficult to successfully integrate replacement genes into the genome with the fast divisional nature of cells. Currently, patients must receive injections and therapy for life, but even then the defect is only solved in isolation and will still be passed down through reproduction. In this way hereditary diseases are not eliminated, only subdued in individual cases.

Unintended mutation

It is always possible for replacement genes to mutate and the carriers, the viral vectors, to regain the ability to act harmful as viruses. In this case gene therapy would have worsened the problem and possibly created more.

Gene placement

Another potential cause for gene replacement backfire is that if the gene does not get inserted into the correct place in the DNA strand. This will also cause new problems.


Whenever a foreign body is introduced into the body it is at risk of rejection. If a replacement gene were to be attacked by the immune system, not only would it have serious and immediate physical repercussions but the body would also build up immunity to the replacement gene and make it impossible for that specific treatment to be used again in the patient.

Gene disorder complexity

Some genetic disorders such as ADA deficiency are the simple cause of a single gene mutation. However, most of the more common genetic diseases such as heart disease, high blood pressure, Alzheimer's, arthritis, diabetes and cancer are all caused by a myriad of different gene mutations. It is extremely very difficult to diagnose these complex problems and tell what modifications need to be done.


There is also the problem that the required replacement gene is not available. This obviously makes treatment of specific gene mutations impossible.

As we can see, gene therapy and modification is not an easy task. It is an incredibly complicated science the deals with our most intricate systems. Whether it's sport or disease related diagnosis and treatment is very complex, and we should be thankful for all the past and similarly difficult advancements that have been made to benefit our lives and civilization.

See also Gene Therapy

And Ethics of Gene Therapy